Compassionate use, also called “expanded access,” provides a pathway for patients with serious diseases to gain access to drugs that are being tested, but have not yet been approved by the U.S. Food and Drug Administration. These drugs are normally available only to people who are taking part in a clinical trial. Being able to use one of these drugs when you are not in a clinical trial is referred to as compassionate drug use.
Access to investigational treatments requires not only FDA’s review and authorization but also the active involvement and cooperation of other parties, including health care providers and the drug company.
In addition to Compassionate Use, the Right to Try Act of 2017 provides a federal framework for certain patients to access certain investigational new drug products that have completed a Phase I clinical trial and that are undergoing investigation for FDA approval. Under certain circumstances, eligible patients can seek treatment without enrolling in clinical trials and without obtaining FDA permission under the FDA expanded access program. There is no obligation for a pharmaceutical manufacturer to make its drug products available to eligible patients as a result of the Right to Try Act.